CRIM-negative infantile Pompe disease, long-term observation of the effect of enzyme replacement therapy: a clinical case
Background. Infantile-onset form of Pompe disease (IOPD) comprises a progressive and fatal disease in the absence of pathogenetic treatment. Enzyme replacement therapy using biologically active recombinant human alglucosidase alfa is considered as a treatment option to increase the life expectancy o...
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| Main Authors: | , , , , |
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| Format: | Article |
| Language: | Russian |
| Published: |
Ministry of Healthcare of the Russian Federation. “Kuban State Medical University”
2024-04-01
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| Series: | Кубанский научный медицинский вестник |
| Subjects: | |
| Online Access: | https://ksma.elpub.ru/jour/article/view/3382 |
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